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Title: Optimising iron chelation therapy with deferasirox for non-transfusion-dependent thalassaemia patients: 1-year results from the THETIS study
Authors: Ali T. Taher
M. Domenica Cappellini
Yesim Aydinok
John B. Porter
Zeynep Karakas
Vip Viprakasit
Noppadol Siritanaratkul
Antonis Kattamis
Candace Wang
Zewen Zhu
Victor Joaquin
Marie José Uwamahoro
Yong Rong Lai
American University of Beirut Medical Center
Università degli Studi di Milano
Ege University Medical School
Istanbul Tip Fakultesi
Mahidol University
University of Athens
Novartis Pharmaceuticals
Novartis International AG
Guangxi Medical University
Keywords: Biochemistry, Genetics and Molecular Biology
Issue Date: 1-Mar-2016
Citation: Blood Cells, Molecules, and Diseases. Vol.57, (2016), 23-29
Abstract: © 2015 The Authors. Efficacy and safety of iron chelation therapy with deferasirox in iron-overloaded non-transfusion-dependent thalassaemia (NTDT) patients were established in the THALASSA study. THETIS, an open-label, single-arm, multicentre, Phase IV study, added to this evidence by investigating earlier dose escalation by baseline liver iron concentration (LIC) (week 4: escalation according to baseline LIC; week 24: adjustment according to LIC response, maximum 30 mg/kg/day). The primary efficacy endpoint was absolute change in LIC from baseline to week 52. 134 iron-overloaded non-transfusion-dependent anaemia patients were enrolled and received deferasirox starting at 10 mg/kg/day. Mean actual dose ± SD over 1 year was 14.70 ± 5.48 mg/kg/day. At week 52, mean LIC ± SD decreased significantly from 15.13 ± 10.72 mg Fe/g dw at baseline to 8.46 ± 6.25 mg Fe/g dw (absolute change from baseline, -6.68 ± 7.02 mg Fe/g dw [95% CI: -7.91, -5.45]; P < 0.0001). Most common drug-related adverse events were gastrointestinal: abdominal discomfort, diarrhoea and nausea (n = 6 each). There was one death (pneumonia, not considered drug related). With significant and clinically relevant reductions in iron burden alongside a safety profile similar to that in THALASSA, these data support earlier escalation with higher deferasirox doses in iron-overloaded non-transfusion-dependent anaemia patients.
ISSN: 10960961
Appears in Collections:Scopus 2016-2017

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